Laura A Adang, M.D., PhD
Assistant Professor of Neurology at the Children's Hospital of Philadelphia
Department: Neurology
Contact information
The Children's Hospital of Philadelphia
3400 Civic Blvd.
Philadelphia, PA 19104
3400 Civic Blvd.
Philadelphia, PA 19104
Office: 215-590-1710
Email:
adangl@email.chop.edu
adangl@email.chop.edu
Education:
BS (Interdisciplinary Studies: Neurology - Magna cum laude with Honors)
University of Georgia, 2001.
PhD (Microbiology)
University of Virginia, 2007.
MD
University of Virginia, 2009.
MS (Translational Research )
Penn, 2018.
Permanent linkBS (Interdisciplinary Studies: Neurology - Magna cum laude with Honors)
University of Georgia, 2001.
PhD (Microbiology)
University of Virginia, 2007.
MD
University of Virginia, 2009.
MS (Translational Research )
Penn, 2018.
Description of Clinical Expertise
LeukodystrophiesDescription of Research Expertise
Clinical and biologic outcome measures in the leukodystrophiesSelected Publications
Pham V, Tricoli L, Hong X, Wongkittichote P, Castruccio Castracani C, Guerra A, Schlotawa L, Adang LA, Kuhs A, Cassidy MM, Kane O, Tsai E, Presa M, Lutz C, Rivella SB, Ahrens-Nicklas RC: Hematopoietic stem cell gene therapy improves outcomes in a clinically relevant mouse model of multiple sulfatase deficiency. Mol Therapy 32(11): 3829-3846, November 2024.Kornbluh AB, Baldwin A, Fatemi A, Vanderver A, Adang LA, Van Haren K, Sampson J, Eichler FS, Sadjadi R, Engelen M, Orthmann-Murphy JL.: Practical Approach to Longitudinal Neurologic Care of Adults With X-Linked Adrenoleukodystrophy and Adrenomyeloneuropathy. Neurol Genet 10(5): e200192, October 2024.
Kotes E, Gavazzi F, Woidill S, Sevagamoorthy A, Yang E, Smith V, Dubbs H, Pierce SR, Pucci K, Vithayathil J, Thakur N, Adang LA: Determination of Health Concepts in β-Propeller Protein-Associated Neurodegeneration. Journal of Child Neurology October 2024.
Peixoto de Barcelos I, Jan AK, Modesti N, Woidill S, Gavazzi F, Isaacs D, D'Aiello R, Sevagamoorthy A, Charlton L, Pizzino A, Schmidt J, van Haren K, Keller S, Eichler F, Emrick LT, Fraser JL, Shults J, Vanderver A, Adang LA.: Systemic complications of Aicardi Goutières syndrome using real-world data. Mol Genet Metab 143(1-2), September-October 2024.
Gavazzi F, Gonzalez CD, Arnold K, Swantkowski M, Charlton L, Modesti N, Dar AA, Vanderver A, Bennett M, Adang LA.: Nucleotide metabolism, leukodystrophies, and CNS pathology. J Inherit Metab Dis 47(5): 860-875, September 2024.
Schoenmakers DH, van den Berg S, Timmers L, Adang LA, Bäumer T, Bosch A, van de Casteele M, Datema MR, Dekker H, Donnelly C, Driessens MHE, Graessner H, Greger V, Haddad T, Höglinger GU, van den Hout H, Jonker C, Langeveld M, Lambert LJ, Neacy E, Nieuwland M, Klockgether T, van der Knaap MS, Papadopoulou A, Plueschke K, van Rijn S, Rosenberg N, Saunier-Vivar EF, Dos Santos Vieira B, Hollak CEM, Goettsch WG, Wolf NI.: Framework for Multistakeholder Patient Registries in the Field of Rare Diseases: Focus on Neurogenetic Diseases. Neurology 103(6): e209743, September 2024.
Pham V, Tricoli L, Hong X, Wongkittichote P, Castruccio Castracani C, Guerra A, Schlotawa L, Adang LA, Kuhs A, Cassidy MM, Kane O, Tsai E, Presa M, Lutz C, Rivella SB, Ahrens-Nicklas RC.: Hematopoietic stem cell gene therapy improves outcomes in a clinically relevant mouse model of multiple sulfatase deficiency. Mol Ther 32: 3829-3846, Aug 2024.
Gavazzi F, Vaia Y, Woidill S, Formanowski B, Peixoto de Barcelos I, Sevagamoorthy A, Modesti NB, Charlton L, Cusack SV, Vincent A, D'Aiello R, Jawad A, Galli J, Varesio C, Fazzi E, Orcesi S, Glanzman AM, Lorch S, DeMauro SB, Guez-Barber D, Waldman AT, Vanderver A, Adang LA.: Nonverbal Cognitive Skills in Children With Aicardi Goutières Syndrome. Neurology 103(1): e209541, July 2024.
Adang LA, Bonkowsky JL, Boelens JJ, Mallack E, Ahrens-Nicklas R, Bernat JA, Bley A, Burton B, Darling A, Eichler F, Eklund E, Emrick L, Escolar M, Fatemi A, Fraser JL, Gaviglio A, Keller S, Patterson MC, Orchard P, Orthmann-Murphy J, Santoro JD, Schöls L, Sevin C, Srivastava IN, Rajan D, Rubin JP, Van Haren K, Wasserstein M, Zerem A, Fumagalli F, Laugwitz L, Vanderver A.: Consensus guidelines for the monitoring and management of metachromatic leukodystrophy in the United States. Cytotherapy 26(7): 739-748, July 2024.
Adang LA, D'Aiello R, Takanohashi A, Woidill S, Gavazzi F, Behrens EM, Sullivan KE, Goldbach-Mansky R, de Jesus AA; AGS Clinical Trial Readiness Workgroup; Vanderver A, Shults J.: IFN-signaling gene expression as a diagnostic biomarker for monogenic interferonopathies. JCI Insight 9(14): e178456, June 2024.